eprintid: 1481142
rev_number: 35
eprint_status: archive
userid: 608
dir: disk0/01/48/11/42
datestamp: 2016-04-17 13:44:51
lastmod: 2021-12-13 01:33:00
status_changed: 2016-12-21 11:55:56
type: article
metadata_visibility: show
creators_name: Booth, C
creators_name: Gaspar, HB
creators_name: Thrasher, AJ
title: Treating Immunodeficiency through HSC Gene Therapy
ispublished: pub
divisions: UCL
divisions: B02
divisions: D13
divisions: G24
keywords: Science & Technology, Life Sciences & Biomedicine, Biochemistry & Molecular Biology, Cell Biology, Medicine, Research & Experimental, Research & Experimental Medicine, Chronic Granulomatous-disease, Leukocyte Adhesion Deficiency, Hematopoietic Stem-cells, Adenosine-deaminase-deficiency, Wiskott-aldrich Syndrome, Lentiviral Vector, Lymphoproliferative Disease, Murine Model, X-cgd, Mice
note: Copyright © 2016. This manuscript version is published under a Creative Commons Attribution Non-commercial Non-derivative 4.0 International licence (CC BY-NC-ND 4.0). This licence allows you to share, copy, distribute and transmit the work for personal and non-commercial use providing author and publisher attribution is clearly stated. Further details about CC BY licences are available at http://creativecommons.org/licenses/by/4.0. Access may be initially restricted by the publisher.
abstract: Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a therapeutic option to treat specific inherited immune deficiencies, including severe combined immune deficiencies (SCID) over the past two decades. Initial clinical trials using first-generation gamma-retroviral vectors to transfer corrective DNA demonstrated clinical benefit for patients, but were associated with leukemogenesis in a number of cases. Safer vectors have since been developed, affording comparable efficacy with an improved biosafety profile. These vectors are now in Phase I/II clinical trials for a number of immune disorders with more preclinical studies underway. Targeted gene editing allowing precise DNA correction via platforms such as ZFNs, TALENs and CRISPR/Cas9 may now offer promising strategies to improve the safety and efficacy of gene therapy in the future.
date: 2016-04
date_type: published
publisher: ELSEVIER SCI LTD
official_url: http://dx.doi.org/10.1016/j.molmed.2016.02.002
oa_status: green
full_text_type: other
language: eng
primo: open
primo_central: open_green
article_type_text: Review
verified: verified_manual
elements_id: 1120523
doi: 10.1016/j.molmed.2016.02.002
lyricists_name: Booth, Claire
lyricists_name: Gaspar, Hubert
lyricists_name: Thrasher, Adrian
lyricists_id: CABOO05
lyricists_id: HBGAS19
lyricists_id: ATHRA78
full_text_status: public
publication: Trends In Molecular Medicine
volume: 22
number: 4
pagerange: 317-327
pages: 11
issn: 1471-4914
citation:        Booth, C;    Gaspar, HB;    Thrasher, AJ;      (2016)    Treating Immunodeficiency through HSC Gene Therapy.                   Trends In Molecular Medicine , 22  (4)   pp. 317-327.    10.1016/j.molmed.2016.02.002 <https://doi.org/10.1016/j.molmed.2016.02.002>.       Green open access   
 
document_url: https://discovery.ucl.ac.uk/id/eprint/1481142/1/Booth_Treating%20Immunodeficiency%20through%20HSC%20Gene%20Therapy.pdf
document_url: https://discovery.ucl.ac.uk/id/eprint/1481142/2/Booth_Treating%20Immunodeficiency%20through%20HSC%20Gene%20Therapy%20Figure%201.pdf
document_url: https://discovery.ucl.ac.uk/id/eprint/1481142/3/Booth_Treating%20Immunodeficiency%20through%20HSC%20Gene%20Therapy%20Figure%202.pdf